Cerebral Therapeutics Announces Publication of Phase 2a Clinical Trial Results

Cerebral Therapeutics publishes the first human study to test a long-term anti-seizure therapy through implantation of an intracerebroventricular drug delivery system for drug-resistant epilepsy

AURORA, Colo., June 1, 2020 - Cerebral Therapeutics, a clinical-stage pharmaceutical company developing innovative treatments for uncontrolled neurological diseases, announced today the publication in The Lancet - EClinicalMedicine of results from its Phase 2a clinical trial evaluating intracerebroventricular drug delivery of the anti-seizure medication sodium valproate in subjects with medically refractory epilepsy. 

A Phase 2a clinical study was undertaken over the course of two and a half years at St. Vincent’s Hospital at The University of Melbourne in Australia evaluating the therapy in subjects with drug resistant focal seizures through use of an implantable infusion system to deliver a reformulation of the anti-seizure medication sodium valproate into the cerebrospinal fluid, bypassing the blood-brain barrier. In the study, five adult subjects who were refractory to oral valproate were treated with this therapy in order to evaluate the dose range of sodium valproate administered and to evaluate the safety and efficacy of this therapy. The study found that 80% of subjects were responders (> 50% seizure reduction), and three subjects experienced periods of complete seizure freedom. All five subjects reported significant quality of life improvements, with minimal drug side effects. The study demonstrated that intracerebroventricular administration of sodium valproate appears safe and well tolerated in subjects with medically refractory epilepsy over many months. 

This is the first demonstration of chronic intracerebroventricular therapy as a potentially effective strategy in the treatment of medically resistant subjects. Despite the refractory nature of epilepsy in these subjects and the unsuccessful earlier use of oral valproate, this proprietary infusion therapy is safe, well tolerated, and holds promise as an effective therapy for refractory epilepsy. Safety and efficacy will be further explored in a Phase 2a extension study, as well as an upcoming Phase 2b randomized controlled study.   

The article can be found: https://www.thelancet.com/pdfs/journals/eclinm/PIIS2589-5370(20)30070-5.pdf